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The first patient to use CRISPR to treat sickle cell disease says the technology relieves symptoms

A mother of four, the first American patient to have her genes processed with the controversial CRISPR technology to treat her sickle cell disease, says the technology works.

Victoria Gray, 34, of Forest, Mississippi, has suffered from the debilitating blood disease her whole life.

At one point, she was in so much pain that she was not even able to lift a spoon, let alone take care of her children.

Sickle cell disease affects as many as 100,000 Americans – a disproportionate number of whom are black, such as Gray – and while treatments can relieve symptoms, they do nothing to address the underlying disease.

But the genetically modified cells that doctors have put in her body seem to reverse and relieve the symptoms of the debilitating disease, NPR reported.

Tests show no signs that Gray’s DNA has changed, and she says she no longer experiences the severe pain attacks that should have hospitalized her several times a year.

Victoria Gray, 34 (photo), from Forest, Mississippi, became the first U.S. patient to use CRISPR to treat her sickle cell disease

The gene editing tool created new fetal hemoglobin, the oxygen-carrying protein, to eliminate the defective hemoglobin that caused her red blood cells to stick together in blood vessels.  Shown: gray

The gene editing tool created new fetal hemoglobin, the oxygen-carrying protein, to eliminate the defective hemoglobin that caused her red blood cells to stick together in blood vessels.  Shown: gray

Victoria Gray, 34 (left and right), from Forest, Mississippi, became the first American patient to use CRISPR to treat her sickle cell disease. The gene editing tool created new fetal hemoglobin, the oxygen-carrying protein, to eliminate the defective hemoglobin that caused her red blood cells to stick together in blood vessels

Gray says she no longer suffers from severe pain attacks that would have hospitalized her and required no blood transfusions or narcotics.  In the photo: gray with three of her children

Gray says she no longer suffers from severe pain attacks that would have hospitalized her and required no blood transfusions or narcotics.  In the photo: gray with three of her children

Gray says she no longer suffers from severe pain attacks that would have hospitalized her and required no blood transfusions or narcotics. In the photo: gray with three of her children

‘It’s beautiful. It’s the change I’ve been waiting for all my life, “Gray told NPR.

“It’s hard to put into words the joy I feel – being thankful for such a big change. It was amazing, ‘

Sickle cell disease is an inherited condition that affects the way in which red blood cells circulate oxygen throughout the body.

The condition causes red blood cells to have a crescent or sickle shape instead of the normal round shape.

Sickle cells die prematurely, causing a shortage of red blood cells. When they pass through the blood vessels, they stick together and block the blood vessels, preventing the blood from flowing properly.

The buildup of sickle cells that periodically appear in Grey’s blood vessels often gives her enormous pain.

HOW DOES CRISPR DNA PROCESSING WORK?

The CRISPR gene editing technique is increasingly used in health research because it can change the building blocks of the body.

At a basic level, CRISPR works as a DNA cut and paste operation.

Technically called CRISPR-Cas9, the process involves sending new strands of DNA and enzymes to organisms to edit their genes.

In humans, genes act as blueprints for many processes and features in the body – they dictate everything from the color of your eyes and hair to whether or not you have cancer.

The components of CRISPR-Cas9 – the DNA sequence and the enzymes needed to implant it – are often sent into the body on the back of a harmless virus so that scientists can determine where they are going.

Cas9 enzymes can then cut DNA strands, effectively knock out a gene or remove parts of DNA that need to be replaced by the CRISPRs, new parts that are sent in to change the gene and have an effect for which they are preprogrammed to produce.

But the process is controversial because it could be used to change babies in the womb – initially to treat diseases – but it could lead to an increase in ‘designer babies’ because doctors offer ways to change the DNA of embryos.

Source: Broad institute

According to the Centers for Disease Control and Prevention, the disease is much more common in African Americans, with an estimated one in 13 born with the trait.

About 365 African American and black babies are born with the disease itself and many will die in their forties.

Treatment mainly focuses on relieving symptoms, such as pain and infections, through blood transfusions and pain killers.

But this new experimental treatment, which Gray received on July 2, 2019, is different.

Scientists removed cells from her bone marrow and modified a gene to make new fetal hemoglobin, the oxygen-carrying protein.

This is usually only produced by fetuses in the womb to draw oxygen from their mother’s blood.

If it works, the fetal protein can be a lifelong solution for Gray and hundreds of thousands more sickle cell patients around the world by compensating for the faulty hemoglobin her body makes.

Blood tests viewed by NPR showed that about 46 percent of the hemoglobin in Gray’s system is fetal hemoglobin, and 99.7 percent of its red blood cells contained part of it.

The team says they would have considered the treatment successful if only 20 percent of the hemoglobin in her system was fetal hemoglobin.

In the past two years, Gray told NPR that because of her pain, she would be hospitalized about seven times a year and would need either regular blood transfusions or prescription narcotics.

Now she has no more severe pain attacks, no hospitalization and no blood transfusions.

“Graduating from high school, graduating from college, weddings, grandchildren – I thought I wouldn’t see any of that,” Gray told the radio station.

“Now I will be there to help my daughters choose their wedding dresses and we will be able to take family vacations and they will have their mother every step of the way.”

Gray's condition means that many of her red blood cells are in the form of sticky sickles (center), rather than being smooth, round and oxygenated discs

Gray's condition means that many of her red blood cells are in the form of sticky sickles (center), rather than being smooth, round and oxygenated discs

Gray’s condition means that many of her red blood cells are in the form of sticky sickles (center), rather than being smooth, round and oxygenated discs

In late 2019, Gray’s husband, who is with the National Guard, was posted to Washington.

She says she doesn’t know how she could have cared for her three children on her own if she hadn’t been for treatment.

“Since my treatment, I have been able to do everything for myself, everything for my children,” Gray told NPR.

“And so it was not only joy for me, but also for the people around me who are in my life.”

While animal and petri dish tests have been successful enough to give the Food and Drug Administration the green light for live human trials, it has never been done before.

It’s possible that CRISPR could cause unwanted DNA changes, at worst, so the scientists say they have to follow Gray for years to file a lawsuit

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